PTC Therapeutics, Inc. (PTCT) announced positive results on two different long-term extension studies on vatiquinone. The company also announced regulatory progress on vatiquinone for the treatment of Friedreich ataxia (FA). Following the announcement, PTCT stock gained 16.52%. Image Source: Zacks Investment Research Year to date, shares of PTC Therapeutics have risen 48% against the industry's 4.6% decline. PTCT's Vatiquinone Positive in Long-Term Analysis PTCT announced that the pre-specified endpoint for ...

Financial Data and Key Metrics - Q2 2024 total revenue was $187 million, with DMD franchise revenue contributing $118 million [4] - Translarna net product revenue for Q2 was $70 million, while EMFLAZA net product revenue was $47 million [17] - Royalty revenue from Evrysdi was $53 million, driven by Roche's global revenue of $535 million [17] - Non-GAAP R&D expense was $123 million, down from $170 million in Q2 2023, reflecting strategic portfolio prioritization [17] - Non-GAAP SG&A expense was $60 million, compared to $75 million in Q2 2023 [17] - Cash, cash equivalents, and marketable securities totaled $1.09 billion as of June 30, 2024, up from $877 million at the end of 2023 [18] Business Line Performance - DMD franchise delivered $118 million in Q2, with Translarna commercialization ongoing in Europe and international markets [11] - EMFLAZA net revenue was $47 million in Q2, supported by patient programs and strong payer relationships [12] - Sepiapterin NDA submitted to the FDA for PKU treatment, with plans for global launch in 2025 [6] - Translarna NDA resubmitted to the FDA, with positive results from Study 41 supporting the submission [7] - PTC518 for Huntington's disease showed promising 12-month interim data, with dose-dependent lowering of mutant Huntington protein and favorable clinical effects [7][8] Market Performance - Translarna commercialization continues in Europe and international markets, with new group purchase orders in Brazil [11] - Sepiapterin launch preparations are underway, with submissions planned for Japan, Brazil, and other countries in 2024 [6][12] - Upstaza received FDA priority review with a target action date of November 13, 2024, and global access discussions are progressing [15] - TEGSEDI and WAYLIVRA showed growth in Latin America, with recent approval in Mexico and new group purchase orders in Brazil [16] Strategic Direction and Industry Competition - Focus on execution and achieving regulatory milestones, including NDA submissions for sepiapterin, Translarna, and Vatiquinone [4][9] - Sepiapterin is positioned as a potential new standard of care for PKU, with data supporting differentiation from current therapies [13] - PTC518 is seen as a promising therapy for Huntington's disease, with plans to pursue accelerated approval based on biomarker and clinical data [8][29] - Utreloxastat for ALS is expected to address significant unmet needs, with top-line results from the CARDINALS trial anticipated in Q4 2024 [9][40] Management Commentary on Operating Environment and Future Outlook - Management highlighted strong execution across all areas, with solid revenue performance and effective expense management [4] - The company updated 2024 revenue guidance to $700 million to $750 million, driven by sustained Translarna revenue in Europe [16] - Management expressed confidence in the potential of sepiapterin, PTC518, and Vatiquinone to address unmet medical needs and drive future growth [6][8][9] Other Important Information - The company completed the sale of its gene therapy manufacturing business in Hopewell, New Jersey, receiving $27.5 million upfront and reducing operational expenses [5] - PTC amended its agreement with Royalty Pharma, exercising a put option for $250 million in cash [18] Q&A Session Summary Question: Huntington's Disease Biomarkers and Safety - PTC518 has shown no evidence of NFL spikes, indicating a favorable safety profile, with dose-dependent lowering of mutant Huntington protein and early clinical benefits [20][21][22] Question: PKU Launch Expectations - Sepiapterin launch is expected to have a fast uptake, particularly in the US, due to well-defined patient identification through newborn screening and strong preparation [23][24] Question: Revenue Guidance and Regulatory Path for PTC518 - Revenue guidance was updated due to sustained Translarna revenue in Europe, with the majority of revenue coming from PTC-marketed products [31] - PTC is preparing for FDA discussions on accelerated approval for PTC518, with plans to submit a Phase III efficacy trial design [26][29] Question: Translarna Regulatory Status in Europe and US - Translarna reexamination in Europe is ongoing, with a focus on addressing concerns around the STRIDE analysis [33] - Translarna NDA resubmission in the US is under review, with expectations of regulatory action within six months [34] Question: ALS Trial Expectations - The CARDINALS trial for utreloxastat in ALS is designed to support NDA submission, with success defined as statistically significant primary endpoint results [39][40] Question: Huntington's Disease Biomarker Correlation - Huntington protein lowering is the most reliable biomarker for clinical benefit, with evidence supporting its use as a surrogate endpoint for accelerated approval [43][44] Question: ALS Trial Design and FDA Expectations - The CARDINALS trial design aligns with FDA expectations, incorporating ALSFRS scale changes and time to death as key endpoints [47] Question: Translarna US Market Opportunity - Translarna is expected to have a rapid uptake in the US, driven by high unmet medical need and strong physician and patient demand [52][53][54] Question: SG&A Expenses and Vatiquinone Data - No significant increase in SG&A expenses is expected for 2025, as the company has built infrastructure to support multiple launches [58][59] - Vatiquinone data from the MOVE FA study will be shared once the long-term analysis is completed [60] Question: Huntington's Disease Surrogate Biomarkers - PTC believes Huntington protein lowering fits FDA guidance for surrogate endpoints, with strong scientific evidence supporting its use [64][65]

PTC Therapeutics (PTCT) came out with a quarterly loss of $1.16 per share versus the Zacks Consensus Estimate of a loss of $1. This compares to loss of $2.66 per share a year ago. These figures are adjusted for nonrecurring items. This quarterly report represents an earnings surprise of -16%. A quarter ago, it was expected that this biopharmaceutical company would post a loss of $1.21 per share when it actually produced a loss of $1.20, delivering a surprise of 0.83%. Over the last four quarters, the compan ...

Financial Performance - Translarna generated $70.4 million in net sales during the quarter ended June 30, 2024[179]. - Emflaza achieved $47.3 million in net sales for the same quarter[179]. - Net product revenues for Q2 2024 were $133.2 million, a decrease of $41.4 million, or 24%, from $174.6 million in Q2 2023, primarily due to lower sales of Translarna and Emflaza[234]. - Emflaza net product revenues decreased by $18.4 million, or 28%, to $47.3 million in Q2 2024, driven by the expiration of orphan drug exclusivity[235]. - Translarna net product revenues were $70.4 million in Q2 2024, down $26.1 million, or 27%, compared to $96.5 million in Q2 2023, attributed to the timing of bulk government orders[235]. - Net product revenues for the six months ended June 30, 2024, were $310.8 million, a decrease of $51.3 million, or 14%, from $362.1 million in the same period of 2023[247]. - Net product sales outside of the United States for the three months ended June 30, 2024, were $85.9 million, a decrease of 21.1% compared to $108.9 million in the same period of 2023[212]. - Net product sales in the United States for the three months ended June 30, 2024, were $47.3 million, down 28.0% from $65.7 million in the same period of 2023[212]. Regulatory Developments - The marketing exclusivity period for Emflaza related to DMD in patients aged five years and older expired in February 2024, expected to negatively impact revenue[186]. - Upstaza received approval from the EC for AADC deficiency treatment in July 2022 and the FDA accepted its BLA for review with a target action date of November 13, 2024[189]. - The CHMP issued a negative opinion on the renewal of Translarna's conditional marketing authorization, which is currently under re-examination[182]. - The company plans to submit regulatory applications for sepiapterin in Japan and Brazil later in 2024[195]. - The MAA submission for sepiapterin was validated by the EMA, triggering a $15.0 million regulatory milestone payment[207]. - A BLA for gene therapy for AADC deficiency was submitted to the FDA in March 2024, with priority review granted and a target action date of November 13, 2024[275]. - The MAA for sepiapterin for PKU was validated by the EMA in May 2024, triggering a $15 million regulatory milestone payment[280]. - The company expects to make additional payments of $50 million upon achieving regulatory milestones related to sepiapterin in 2024[280]. - As of June 30, 2024, potential remaining regulatory milestones for Upstaza are estimated at $11.1 million, with potential sales milestones of $50 million[280]. Research and Development - Sepiapterin demonstrated a 63% reduction in blood Phe levels in the primary analysis population during its Phase 3 trial for phenylketonuria[192]. - PTC518 demonstrated a mean 30% reduction in mutant HTT levels at the 10mg dose level during the Phase 2 study for Huntington's disease[196]. - Vatiquinone showed significant benefits on key disease subscales and secondary endpoints in the MOVE-FA trial, despite not meeting the primary endpoint[197]. - The company plans to submit an NDA for vatiquinone in late 2024 based on the MOVE-FA trial data[197]. - Utreloxastat was well-tolerated in a Phase 1 trial, and a Phase 2 trial for ALS is expected to yield topline results in Q4 2024[197]. - Research and development expenses for the three months ended June 30, 2024, were $132.2 million, a decrease of 29.0% from $185.9 million in the same period of 2023[225]. - Total research and development expenses for the six months ended June 30, 2024, were $248.3 million, down 34.8% from $381.0 million in the same period of 2023[225]. - Research and development expense was $248.3 million for the six months ended June 30, 2024, a decrease of $132.7 million, or 35%, from $381.0 million for the same period in 2023[254]. Financial Position and Cash Flow - The company has an accumulated deficit of $3,474.3 million as of June 30, 2024, with a net loss of $190.8 million for the six months ended June 30, 2024[204]. - As of June 30, 2024, the company had cash, cash equivalents, and marketable securities of $1.09 billion[270]. - Net cash used in operating activities was $0.7 million for the six months ended June 30, 2024, compared to $43.6 million for the same period in 2023, primarily due to clinical development and commercial activities, offset by a $100 million sales milestone from Evrysdi[272]. - Net cash used in investing activities increased to $180.9 million for the six months ended June 30, 2024, from $52.7 million in the same period of 2023, mainly for product rights acquisition and marketable securities[273]. - Net cash provided by financing activities rose significantly to $247.0 million for the six months ended June 30, 2024, compared to $22.6 million in 2023, driven by proceeds from sales of future royalties and employee stock purchase plans[274]. Operational Challenges and Future Outlook - The company expects to incur significant expenses related to commercialization efforts and ongoing clinical trials[205]. - The company may seek to expand its product pipeline through in-licensing or acquisitions, which could increase future capital requirements[206]. - The company anticipates ongoing significant expenses related to commercialization efforts in various regions, including the U.S. and EEA, and costs for clinical trials and studies for sepiapterin and other programs[275]. - The company has never been profitable and will need to generate significant revenues to achieve and sustain profitability, potentially requiring substantial additional funding[282]. - If unable to raise additional funds, the company may need to delay or reduce product development and commercialization efforts[283].

Exhibit 99.1 PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2024 Financial Results – Continued strong revenue performance – – NDAs for sepiapterin and Translarna™ submitted to FDA – – Positive interim data readout from PTC518 PIVOT-HD study – – On track to achieve remaining 2024 clinical and regulatory milestones – WARREN, N.J., August 8, 2024 – PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ending June 30, 2024. ...

Analysts on Wall Street project that PTC Therapeutics (PTCT) will announce quarterly loss of $1 per share in its forthcoming report, representing an increase of 62.4% year over year. Revenues are projected to reach $192.12 million, declining 10.1% from the same quarter last year. The consensus EPS estimate for the quarter has undergone an upward revision of 2% in the past 30 days, bringing it to its present level. This represents how the covering analysts, as a whole, have reassessed their initial estimates ...

WARREN, N.J., July 30, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the sepiapterin NDA to the U.S. FDA. The NDA submission is for the treatment of pediatric and adult patients with phenylketonuria (PKU), including the full spectrum of ages and disease subtypes. "This NDA submission is an important step in bringing this therapy to children and adults living with PKU in the U.S.," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The ro ...

Investors: Kylie O'Keefe +1 (908) 300-0691 [email protected] SOURCE PTC Therapeutics, Inc. WARREN, N.J., July 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will host a webcast conference call to report its second quarter 2024 financial results and provide an update on the company's business and outlook on Thursday, Aug. 8, at 4:30 p.m. EDT. To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid dela ...

While the sustainability of the immediate price change and future earnings expectations will mostly depend on management's discussion of business conditions on the earnings call, it's worth handicapping the probability of a positive EPS surprise. Estimate Revisions Trend Earnings Whisper A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive s ...

NEW YORK, July 09, 2024 (GLOBE NEWSWIRE) -- Attorney Advertising -- Bronstein, Gewirtz & Grossman, LLC is investigating potential claims on behalf of purchasers of PTC Therapeutics, Inc. ("PTC" or "the Company") (NASDAQ: PTCT). Investors who purchased PTC securities are encouraged to obtain additional information and assist the investigation by visiting the firm's site: bgandg.com/PTCT. Investigation Details On June 28, 2024, PTC issued a press release announcing that the European Medicines Agency's Committ ...